Ruxoprubart

--- Ruxoprubart (NM8074) met all clinical endpoints, offering a safe, differentiated treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological (Blood) disorder. Regulatory approval for the Phase II trial in PNH subjects was granted based on the safety profile of healthy subjects in the Phase I trial. Interim results from this 12-subject, three-month, multi-dose Phase II efficacy trial are compelling. The drug was safe and well-tolerated in all treated PNH subjects with expected safety & efficacy, meeting all clinical endpoints with no reported side effects. In this efficacy trial, Ruxoprubart: Protected PNH Red Blood cells against lysis and enabled complete transfusion avoidance, meeting the primary endpoint. Increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject hemoglobin increase of 1.5 to 4.3 g/dL, exceeding the threshold set for the primary endpoint. An average hemoglobin increase of >1.6 g/dL was observed across the cohort from baseline to the end of the treatment period. Reduced LDH levels to baseline, meeting the primary efficacy endpoint. Significantly increased PNH Type III clone size by protecting PNH Red Blood Cells from complement-mediated lysis. Allowed normal pathogenic clearance in all treated PNH subjects, as infection cleared as expected. The regulatory agency has recently approved a once-weekly subcutaneous protocol for Ruxoprubart — allowing for self-administration and offering a distinct advantage over Apellis, which requires twice-weekly dosing. Ruxoprubart effectively inhibited the Alternative Pathway (AP) without affecting the Classical Pathway (CP) in the trial. More importantly, the FDA has granted Orphan Drug Designation (ODD) to Ruxoprubart for treating PNH. A Breakthrough Therapy Designation (BTD) application for Ruxoprubart in PNH will soon be filed for FDA review. Ruxoprubart is set to be developed as a novel treatment for several complement-mediated disorders, including but not limited to renal, hematological, dermatological, and ocular diseases. CLEVELAND, May 19, 2025 (GLOBE NEWSWIRE) -- NovelMed is pleased to announce positive 12-week interim results from the ongoing multi-dose Phase II trial of Ruxoprubart, a novel complement-targeting immunotherapy, in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). These interim data demonstrate that Ruxoprubart, administered as monotherapy, was safe, well-tolerated, and met all primary efficacy endpoints. Ruxoprubart achieved transfusion avoidance, increased hemoglobin levels, reduced LDH, and increased PNH clone size — key outcomes that contributed to improved disease control and enhanced quality of life for PNH patients. “The promising interim results from our Phase II PNH study solidify Ruxoprubart as a novel treatment for patients with advanced hematological conditions, offering hope where existing therapies fall short. This achievement not only underscores our commitment to advancing Ruxoprubart, an immunotherapy for PNH, but also marks a significant step toward addressing critical unmet needs within this patient population," said Dr. Rekha Bansal, CEO. "I extend my sincere gratitude to the PNH subjects and investigators whose dedication has been instrumental in this success. We are eager to present our findings to regulatory authorities and proceed with the Phase III trial." Figure. NovelMed Scientist Prepares Complement Test Assays to Support Clinical Development of Ruxoprubart (NM8074) for Paroxysmal Nocturnal Hemoglobinuria (PNH). Ruxoprubart works by binding to Bb and selectively inhibiting the protease activity of the Alternative Pathway C3 convertase, while preserving the Classical Pathway. By acting proximally in the Alternative Pathway, Ruxoprubart provides complete control over both intravascular and extravascular hemolysis, resulting in marked increases in hemoglobin levels observed in this trial. These findings support the drug’s advancement as a novel, next-generation immunotherapy for the treatment of PNH and a broad range of hematological, renal, inflammatory, and ocular diseases. “We are excited to announce that our Alternative Pathway strategy for treating PNH is delivering exceptional results,” said Mr. Alex Kumar, Chief Strategy Advisor at NovelMed. “These data reinforce our confidence as we look forward to working with the regulatory agency to bring Ruxoprubart to PNH patients without delay. Our mission is to transform the lives of chronically ill patients for whom an ideal treatment has yet to be discovered.” Clinical Study Overview and Efficacy Summary in PNH This Phase II trial was initiated following the successful completion of a Phase I study involving 40 (forty) healthy volunteers. Ruxoprubart was well tolerated across all dose levels, ranging from 0.3 to 20 mg/kg, with no safety concerns. The drug demonstrated selective inhibition of the Alternative Pathway through Bb binding, without affecting the Classical Pathway. These promising safety findings paved the way for regulatory approval to initiate a Phase II study in patients with PNH. This ongoing 12-week clinical trial of Ruxoprubart is an open-label, multi-dose study in treatment-naïve adult subjects with PNH. The primary objectives are to evaluate the safety, tolerability, and efficacy of Ruxoprubart as a monotherapy in the Alternative Pathway-mediated lysis of Red Blood Cells. To date, 10 out of 12 PNH subjects have completed the study. Subjects 11 & 12 have been enrolled to complete the two-cohort trial. Interim results confirm that Ruxoprubart achieved all primary efficacy endpoints, delivering sustained clinical benefit in PNH. The primary efficacy endpoints include: Transfusion Avoidance : PNH patients typically require multiple blood transfusions to maintain normal hemoglobin levels. Ruxoprubart effectively protected PNH Type III cells from Alternative Pathway-mediated destruction, resulting in complete transfusion avoidance and successfully meeting the primary efficacy endpoint. Hemoglobin Increase : PNH patients experience reduced hemoglobin levels, leading to severe anemia. Ruxoprubart increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject increases ranging from 1.5 to 4.3 g/dL, surpassing the efficacy threshold defined for the primary endpoint. An average increase of >1.6 g/dL in hemoglobin was observed across the cohort from the beginning to the end of the treatment period. LDH Reduction : PNH patients experience severe lysis of Red Blood Cells (RBCs), leading to elevated lactate dehydrogenase (LDH) levels. By controlling complement-mediated hemolysis, Ruxoprubart reduced LDH levels to baseline, achieving the primary efficacy endpoint. Preservation of PNH Cells : Continuous lysis of Red Blood Cells in PNH leads to a dramatic reduction in total RBC count, often exceeding 80 to 90%. By effectively preventing this lysis, Ruxoprubart significantly preserved PNH cells, resulting in a substantial increase in the total PNH clone size and hemoglobin, successfully meeting the primary efficacy endpoint. Pathogenic Clearance : As expected, Ruxoprubart facilitated effective pathogenic clearance across all treated PNH subjects. In summary, the observed changes in efficacy parameters support the drug's mechanism of action and validate Ruxoprubart’s potential as a highly effective monotherapy for PNH. “We are thrilled to share these data, which demonstrate the potential of Ruxoprubart in advanced PNH, and look forward to working with regulatory authorities to bring this new immunotherapy to this patient community,” said Mr. Robert Bard, Vice President of Regulatory Affairs at NovelMed. “It’s very clear that we are on a promising path with exciting Phase II data in this serious hematological disorder.” Paroxysmal Nocturnal Hemoglobinuria (PNH) PNH is a rare, acquired hematologic disorder in which Red Blood Cells lack protective surface proteins due to a mutation in the phosphatidylinositol glycan class A (PIGA) gene, leaving them vulnerable to complement-mediated destruction. In healthy individuals, Type I RBCs express complement regulatory proteins and are fully protected from complement-mediated destruction. In contrast, PNH patients have a significant percentage of Type III RBCs, which completely lack these protective proteins and are therefore highly susceptible to Alternative Pathway-mediated lysis. PNH is primarily an Alternative Pathway-mediated disease, with no involvement of the Classical or Lectin pathways in its pathology. Key laboratory markers indicative of disease progression include reduced Type III Red Blood Cells, low hemoglobin, elevated lactate dehydrogenase (LDH), and an increased need for blood transfusions. These markers reflect the underlying hemolytic anemia, which in turn leads to debilitating symptoms such as chronic fatigue, pain, jaundice, and organ damage. As the disease progresses, it significantly affects the patient’s quality of life and can ultimately result in life-threatening complications and death if not properly managed. While Soliris® (eculizumab) is the current FDA-approved standard of care for PNH – administered via intravenous infusion to provide C5 blockade – clinical limitations persist. Up to 88% of Soliris-treated patients continue to experience persistent anemia, with more than one-third of them requiring blood transfusions at least once every year. Despite its effectiveness in some patients, Soliris does not fully address the needs of many patients, as a significant proportion remain anemic, fatigued, and require blood transfusions, highlighting the need for specific, effective, and comprehensive therapies. Nearly all FDA-approved therapies for PNH and other complement-mediated disorders, oral or intravenous, suffer from the well-documented Black Box warning label because of the broad immunosuppression of the Classical Pathway, elevating the risk of life-threatening infections. Ruxoprubart offers a selective, upstream, and targeted approach to Alternative Pathway inhibition, without compromising the Classical Pathway, offering the best-in-class novel therapy. These attributes position Ruxoprubart as a safer and more effective treatment for patients with PNH. Ruxoprubart (NM8074) Ruxoprubart, an engineered human immunoglobulin, exhibits dual specificity: 1) it blocks only the Alternative Pathway, dysregulated by the disease, and 2) it binds Bb which has no binding to Factor B. Ruxoprubart met all primary endpoints in the interim evaluation of the ongoing Phase II clinical trial in PNH, positioning it as the first immunotherapy in this indication with the potential for approval without a Black Box warning. This immunotherapy has demonstrated a safe and well-tolerated profile across all five cohorts in the Phase I trial, including the highest dose of 20 mg/kg. Our findings confirm that the drug selectively blocks the Alternative Pathway. This differentiated, novel mechanism sets Ruxoprubart apart from other complement blockers (Soliris®, Ultomiris®, Empaveli®, Voydeya®, and Fabhalta®) currently approved or in development worldwide. Voydeya®, which received FDA approval in 2024 as a combination therapy, was developed by Achillion and acquired by AstraZeneca for $930 million. Despite its limited clinical efficacy, Voydeya®'s reliance on co-administration with a C5 inhibitor restricts its potential as a standalone therapy. Unlike Fabhalta®, which targets Factor B, Ruxoprubart (NM8074) binds to Bb, which is generated only upon activation of the Alternative Pathway, offering a unique mechanism of action as monotherapy. As a novel monotherapy with a unique, upstream, and pathway-specific mechanism, Ruxoprubart is emerging as a best-in-class candidate for the treatment of PNH and numerous complement-mediated diseases. NovelMed Therapeutics ( www.novelmed.com ) NovelMed is a clinical-stage biopharmaceutical company dedicated to developing innovative novel biologics (immunotherapies) to treat a wide range of complement-mediated diseases. NovelMed proudly leads the development of Alternative Pathway-specific antibodies (NM8074, NM5072, and NM8070) to address a wide range of chronic complement-mediated disorders. The company is actively conducting a Phase II clinical trial in treatment-naïve PNH subjects to expedite the availability of this promising treatment. Additionally, the FDA has approved Phase II studies for several rare diseases, including but not limited to Atypical Hemolytic Uremic Syndrome (aHUS), C3 Glomerulopathy (C3G), IgA Nephropathy (IgAN), ANCA-Associated Vasculitis (AAV), and Dermatomyositis (DM). We are committed to delivering life-changing therapies to patients across hematology, ophthalmology, nephrology, dermatology, and neurology. NovelMed is currently seeking partnerships to advance the development of Ruxoprubart to approval in several chronic diseases. We are open to discussions regarding investment, out-licensing, or acquisition. For more details regarding our progress, please visit www.novelmed.com/news . Watch our introductory Video to learn more about NovelMed. Contact: Ya Gao, MS NovelMed Communication Team BD@novelmed.com (216) 440 2696 Figure. NovelMed Scientist Prepares Clinical Trial Samples for Analysis https://www.globenewswire.com/NewsRoom/AttachmentNg/ae2c0bfc-ff88-486d-a796-f2977947c8a4 Figure. NovelMed Scientist Prepares Clinical Trial Samples for Analysis Figure. NovelMed Scientist Prepares Clinical Trial Samples for Analysis (Available at https://www.globenewswire.com/NewsRoom/AttachmentNg/ae2c0bfc-ff88-486d-a796-f2977947c8a4) Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. 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EMPAVELI's unique mechanism of targeting C3 broadens its applicability beyond PNH, including other complement-driven diseases like geographic atrophy and immune complex-mediated diseases. With a high unmet need in these areas and limited competition, EMPAVELI's market is poised for steady growth. LAS VEGAS, March 18, 2025 /PRNewswire/ -- DelveInsight's " EMPAVELI Market Size, Forecast, and Market Insight Report" highlights the details around EMPAVELI, a complement inhibitor indicated to treat adult patients with PNH. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of EMPAVELI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Apellis Pharmaceutical's EMPAVELI (pegcetacoplan) Overview EMPAVELI (pegcetacoplan) is a targeted inhibitor of C3 designed to control excessive complement activation—a key immune process that can contribute to the development and progression of serious diseases. Pegcetacoplan binds to complement protein C3 and its activation fragment C3b, helping to regulate the cleavage of C3 and the production of downstream effectors involved in complement activation. In PNH, extravascular hemolysis (EVH) is driven by C3b opsonization, while intravascular hemolysis (IVH) results from the formation of the membrane attack complex (MAC). By acting early in the complement cascade, pegcetacoplan controls both C3b-driven EVH and terminal complement-mediated IVH. EMPAVELI is approved for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). The US FDA has granted pegcetacoplan Priority Review and Fast Track Designation (FTD) for the treatment of PNH. EMPAVELI reported US net product revenue of $23.4 million in the fourth quarter and $98.1 million for the full year of 2024. Currently, EMPAVELI is being evaluated in different phases of development for C3G & IC-MPGN, HSCT-TMA, FSGS, and DGF. Learn more about EMPAVELI projected market size for PNH @ EMPAVELI Market Potential Paroxysmal nocturnal hemoglobinuria (PNH) is a rare condition characterized by a triad of symptoms: intravascular hemolysis, thromboembolic events, and cytopenia. The disease presents differently among patients, making it difficult to classify based on typical clinical patterns due to its unpredictable nature. In 2023, there were approximately 12,000 diagnosed cases of PNH in the 7MM, with projections suggesting this number could rise to around 13,000 by 2034. Until 2007, PNH was a life-threatening disease with no effective treatment for hemolysis and thrombosis, which were the primary causes of death. However, the introduction of the anti-C5 agent eculizumab over the past decade marked a major breakthrough, significantly reducing hemolysis, decreasing the need for transfusions, and lowering the risk of thrombosis. The current standard of care for PNH involves complement inhibition therapy, with FDA-approved drugs like SOLIRIS, ULTOMIRIS, EMPAVELI, FABHALTA, VOYDEYA, and PIASKY being considered the gold standard treatments. According to DelveInsight, the total PNH market size in the 7MM was valued at USD 1.4 billion in 2023 and is expected to grow to about USD 2.5 billion by 2034. This growth is driven by advances in understanding disease mechanisms, leading to improved diagnostics and therapeutics, along with an increasing number of cases and the introduction of new treatments during the forecast period. Discover more about the paroxysmal nocturnal hemoglobinuria market in detail @ Paroxysmal Nocturnal Hemoglobinuria Market Report Emerging Competitors of EMPAVELI Only a few companies are currently working in the paroxysmal nocturnal hemoglobinuria market. Emerging PNH therapies include Pozelimab (REGN3918) + Cemdisiran (Regeneron Pharmaceuticals), OMS906 (Omeros), NM8074 (ruxoprubart) (NovelMed), and others, reflecting a dynamic evolution in treatment strategies. In December 2024, Omeros Corporation announced that two posters on zaltenibart (OMS906), its investigational MASP-3 inhibitor and a key activator of the alternative complement pathway, were presented yesterday at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego. The posters focused on zaltenibart's use in treating paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder, and highlighted positive Phase II clinical data and clinical pharmacology analyses that support dose selection for the upcoming Phase III trials in PNH. Enrollment for the Phase III trials is expected to begin in early 2025. To know more about the number of competing drugs in development, visit @ EMPAVELI Market Positioning Compared to Other Drugs Key Milestones of EMPAVELI In October 2023, Apellis Pharmaceuticals, Inc. announced that the FDA had approved the EMPAVELI Injector. In May 2021, Apellis Pharmaceuticals, Inc. announced that the FDA has approved EMPAVELI (pegcetacoplan), the first and only therapy targeting C3 for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). In October 2020, Apellis Pharmaceuticals and Swedish Orphan Biovitrum AB announced a strategic collaboration to accelerate the advancement of systemic pegcetacoplan. In February 2019, Apellis Pharmaceuticals announced that the FDA has awarded Fast Track designation to APL-2, the company's innovative complement factor C3 inhibitor, for treating patients with paroxysmal nocturnal hemoglobinuria (PNH). Discover how EMPAVELI is shaping the PNH treatment landscape @ EMPAVELI Paroxysmal Nocturnal Hemoglobinuria EMPAVELI Market Dynamics EMPAVELI (pegcetacoplan) is a targeted complement inhibitor developed by Apellis Pharmaceuticals for the treatment of PNH. Approved by the FDA in May 2021, EMPAVELI became the first and only C3 inhibitor available for PNH, providing a new mechanism of action compared to existing therapies like C5 inhibitors (eculizumab and ravulizumab). By targeting C3 rather than C5, EMPAVELI addresses both intravascular and extravascular hemolysis, offering broader control over the disease and reducing the need for red blood cell transfusions in patients who remain anemic despite C5 inhibition. This differentiation has positioned EMPAVELI as a compelling alternative for patients with suboptimal responses to C5 inhibitors. The market for EMPAVELI is driven by the growing prevalence of PNH, increased awareness of complement pathway disorders, and improved diagnostic capabilities. The PNH market was historically dominated by C5 inhibitors such as Alexion's SOLIRIS (eculizumab) and ULTOMIRIS (ravulizumab), which set a high pricing benchmark and established market acceptance for premium-priced orphan drugs. EMPAVELI's entry has introduced competition, particularly for patients who experience breakthrough hemolysis or insufficient control on C5 therapy. Its ability to offer a more comprehensive mechanism of action and potential for improved patient outcomes has driven adoption, although switching patients from established therapies presents a challenge due to physician familiarity and the entrenched market position of C5 inhibitors. Pricing and reimbursement dynamics also play a significant role in EMPAVELI's market performance. As a high-cost orphan drug, EMPAVELI's adoption is influenced by payer coverage and reimbursement policies, especially in markets with strict cost-control measures. Apellis has strategically positioned EMPAVELI through patient assistance programs and value-based agreements to facilitate market access. Additionally, the subcutaneous administration of EMPAVELI (versus intravenous administration for C5 inhibitors) enhances patient convenience, supporting patient preference and potentially improving adherence. Competitive pressures are also evolving as other complement inhibitors targeting C3, C5, and alternative pathways are under development. For example, Novartis and AstraZeneca are advancing next-generation C5 inhibitors with extended dosing intervals, and other biotech firms are exploring factor D and MASP-2 inhibitors, which could further fragment the complement inhibitor landscape. However, EMPAVELI's first-mover advantage in the C3 inhibition space and its differentiated clinical profile provide a strong foothold in the market. Expansion into additional indications beyond PNH, such as cold agglutinin disease (CAD) and geographic atrophy (GA), could further strengthen EMPAVELI's market position and drive future growth. Dive deeper to get more insight into EMPAVELI's strengths & weaknesses relative to competitors @ EMPAVELI Market Drug Report Table of Contents Related Reports Paroxysmal Nocturnal Hemoglobinuria Market Paroxysmal Nocturnal Hemoglobinuria Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key PNH companies including Hoffmann-La Roche, Alexion Pharmaceuticals, Novartis, Regeneron Pharmaceuticals, BioCryst Pharmaceuticals, among others. Paroxysmal Nocturnal Hemoglobinuria Pipeline Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key paroxysmal nocturnal hemoglobinuria companies, including Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, among others. Aplastic Anemia Market Aplastic Anemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key aplastic anemia companies, including Pfizer, BioLineRx Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, among others. Aplastic Anemia Pipeline Aplastic Anemia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED